Correcting mutations in vitro using CRISPR-Cas9; towards autologous stem cell transplantation in sickle cell disease and X-linked severe combined immunodeficiency

Correcting mutations in vitro using CRISPR-Cas9; towards autologous stem cell transplantation in sickle cell disease and X-linked severe combined immunodeficiency - Correcting SCD and X-SCID causing mutations in vitro using CRISPR-Cas9

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON43429

Enrollment

Registered

2016-04-04

Start date

2016-05-04

Completion date

Unknown

Last updated

2024-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

n.v.t.

Interventions

None listed

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: 1. Presence of either SCD or X-SCID 2. For children: availability of an existing cell line or a planned surgical intervention for patient care reasons 3. Able to provide written permission

Exclusion criteria

Exclusion criteria: none

Design outcomes

Primary

Measure	Time frame
Percentage of cells in which the SCD or X-SCID causing mutations are corrected without detectable mutations in other genes	—

Secondary

Measure	Time frame
n.a.	—

Countries

Netherlands

Outcome results